Prolonged expression of therapeutic levels of human granulocyte colony-stimulating factor in rats following gene transfer to skeletal muscle

Human Gene Therapy

Volumn 7, Issue 12, 1996, Pages 1423-1429

  Bonham, Lynn ^a   Palmer, Theo ^a,b   Miller, A Dusty ^a  

^a Fred Hutchinson Cancer Research Center   (United States)

^b SALK INSTITUTE FOR BIOLOGICAL STUDIES   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

GRANULOCYTE COLONY STIMULATING FACTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ARTICLE; CELL TRANSFORMATION; CONTROLLED STUDY; FEMALE; GENE THERAPY; GENE TRANSFER; HUMAN; HUMAN CELL; ISOGRAFT; MALE; MYOBLAST; NEUTROPENIA; NEUTROPHIL; NONHUMAN; PRESCHOOL CHILD; RAT; SKELETAL MUSCLE; VIRUS RECOMBINANT;

EID: 0029790113     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.1996.7.12-1423     Document Type: Article

References (47)

1. ASANO, S. (1991). Human granulocyte colony-stimulating factor: its basic aspects and clinical applications. Am. J. Ped. Hem. 13, 400-413.
2. ASCADI, G., DICKSON, G., LOVE, D.R., JANI, A., WALSH, F.S., GURUSINGHE, A., WOLFF, J.A., and DAVIES, K.E. (1991). Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs. Nature 352, 815-818.
3. BARR, E., and LEIDEN, J.M. (1991). Systemic delivery of recombinant proteins by genetically modified myoblasts. Science 254, 1507-1509.
4. BARTRAM, C.R., PRZYREMBEL, H., WENDEL, U., BREMER, H.J., SCHAUB, J., and HAAS, J.R. (1981). Glycogenesis type IB complicated by severe granulocytopenia resembling inherited neutropenia. Eur. J. Pediatr. 137, 81-84.
5. BEAUDET, A.L. et al. (1980). Neutropenia and impaired neutrophil migration in type IB glycogen storage disease. J. Pediatr. 97, 906-910.
6. BENNETT, V.J., and CHANG, P.L. (1990). Suppression of immunological response against a novel gene product delivered by implants of genetically modified fibroblasts. Mol. Biol. Med. 7, 471-477.
7. BLAU, H.M., and WEBSTER, C. (1981). Isolation and characterization of human muscle cells. Proc. Natl. Acad. Sci. USA 78, 5623-5627.
8. CHANG, J.M., METCALF, D., GONDA, T.J., and JOHNSON, G.R. (1989). Long-term exposure to retrovirally expressed granulocyte-colony-stimulating factor induces a nonneoplastic granulocytic and progenitor cell hyperplasia without tissue damage in mice. J. Clin. Invest. 84, 1488-1496.
9. COHEN, A.M., ZSEBO, K.M., INOUE, H., HINES, D., BOONE, T.C., CHAZIN, V.R., TSAI, L., RITCH, T., and SOUZA, L.M. (1987). In vivo stimulation of granulopoiesis by recombinant human granulocyte colony-stimulating factor. Proc. Natl. Acad. Sci. USA 84, 2484-2488.
10. COLICELLI, J., and GOFF, S.P. (1986). Isolation of a recombinant murine leukemia virus utilizing a new primer tRNA. J. Virol. 57, 37-45.
11. DAI, Y., ROMAN, M., NAVIAUX, R.K., and VERMA, I.M. (1992). Gene therapy via primary myoblasts: long term expression of factor IX protein following transplantation in vivo. Proc. Natl. Acad. Sci. USA 89, 10892-10895.
12. DALE, D.C., and HAMMOND, W.P. (1988). Cyclic neutropenia: A clinical review. Blood Rev. 2, 178-185.
13. DALE, D.C., BONILLA, M.A., DAVIS, M.W., NAKANISHI, A.M., HAMMOND, W.P., KURTZBERG, J., WANG, W., JAKUBOWSKI, A., WINTON, E., LALEZARI, P., ROBINSON, W., GLASPY, J.A., EMERSON, S., GABRILOVE, J., VINCENT, M., and BOXER, L.A. (1993). A randomized controlled phase III trial of recombinant human granulocyte colony-stimulating factor (Filgrastim) for treatment of severe chronic neutropenia. Blood 81, 2496-2502.
14. DHAWAN, J., PAN, L.C., PAVLATH, G.K., TRAVIS, M.A., LANCTOT, A.M., and BLAU, H.M. (1990). Systemic delivery of human growth factor hormone by injection of genetically engineered myoblasts. Science 254, 1509-1512.
15. FIRE, A., HARRISON, S.W., and DIXON, D. (1990). A modular set of lacZ fusion vectors for studying gene expression in Caenorhabditis elegans. Gene 93, 189-198.
16. FLEISCHMAN, R.A. (1993). Southwestern internal medicine conference: Clinical use of hematopoietic growth factors. Am. J. Med. Sci. 305, 248-273.
17. GREZ, M., AKGUN, E., HILBERG, F., and OSTERTAG, W. (1990). Embryonic stem cell virus, a recombinant murine retrovirus with expression in embryonic stem cells. Proc. Natl. Acad. Sci. USA 87, 9202-9206.
18. HALL-CRAGGS, E.C.B. (1974). Rapid degeneration and regeneration of a whole skeletal muscle following treatment with bupivacaine (Marcaine). Exp. Neurol. 43, 349-358.
19. HAMAMORI, Y. BABRU, S. TIAN, J., and KEDES, L. (1994). Persistent erythropoiesis by myoblast transfer of erythopoietin cDNA. Hum. Gene Ther. 5, 1349-1356.
20. JENKINS, M.K., SCHWARTZ, R.H., and PARDOLL, D.M. (1988). Effects of Cyclosporin A on T cell development and clonal deletion. Science 241, 1655-1658.
21. JIAO, S., SCHULTZ, E., and WOLFF, J.A. (1992a). Intracerebral transplants of primary muscle cells: A potential 'platform' for transgene expression in the brain. Brain Res. 575, 143-147.
22. JIAO, S., WILLIAMS, P., BERG, R.K., HODGEMAN, B.A., LIU, L., REPETTO, G., and WOLFF, J.A. (1992b). Direct gene transfer into nonhuman primate myofibers in vivo. Hum. Gene Ther. 3, 21-33.
23. JIAO, S., GUREVICH, V., and WOLFF, J.A. (1993). Long-term correction of rat model of Parkinson's disease by gene therapy. Nature 362, 450-453.
24. KARPATI, G., POULIOT, Y., ZUBRZYCKA-GAARN, E., CARPENTER, S., RAY, P.N., WORTON, R.G., and HOLLAND, P. (1989). Dystrophin is expressed in mdx skeletal muscle fibers after normal myoblast implantation. Am. J. Pathol. 135, 27-32.
25. LANGE, R.D. (1983). Cyclic hematopoiesis: Human cyclic neutropenia. Exp. Hematol. 11, 435-451.
26. MIDHA, R. et al. (1992). Chronic Cyclosporin A therapy in rats. Microsurgery 13, 273-276.
27. MIGLIACCIO, A.R., MIGLIACCIO, G., DALE, D.C., and HAMMOND, W.P. (1990). Hematopoietic progenitors in cyclic neutropenia: effect of granulocyte colony-stimulating factor in vivo. Blood 75, 1951-1959.
28. MILLER, A.D., and ROSMAN, G.J. (1989). Improved retroviral vectors for gene transfer and expression. Biotechniques 7, 980-990.
29. MILLER, A.D., MILLER, D.G., GARCIA, J.V., and LYNCH, C.M. (1993). Use of retroviral vectors for gene transfer and expression. Methods Enzymol. 217, 581-599.
30. MILLER, D.G., EDWARDS, R.H., and MILLER, A.D. (1994). Cloning of the cellular receptor for amphotropic murine retroviruses reveals homology to that for gibbon ape leukemia virus. Proc. Natl. Acad. Sci. USA 91, 78-82.
31. MORGAN, J.E. (1994). Cell and gene therapy in Duchenne muscular dystrophy. Hum. Gene Ther. 5, 165-173.
32. NICOLA, N.A., METCALF, D., MATSUMOTO, M., and JOHNSON, G.R. (1983). Purification of a factor inducing differentiation in murine myelomonocytic leukemia cells. J. Biol. Chem. 258, 9017-9023.
33. PETERSON, R., KEMPLER, G., and BARKLIS, E. (1991). A stem cell-specific silencer in the primer-binding site of a retrovirus. Mol. Cell. Biol. 11, 1214-1221.
34. RANDO, T.A., and BLAU, H.M. (1994). Primary mouse myoblast purification, characterization, and transplantation for cell-mediated gene therapy. J. Cell Biol. 125, 1275-1287.
35. SALVATORI, G., FERRARI, G., MEZZOGIORNO, A., SERVIDEI, S., COLETTA, M., TONALI, P., GIAVAZZI, R., COSSU, G., and MAVILIO, F. (1993). Retroviral vector-mediated gene transfer into human primary myogenic cells leads to expression in muscle fibers in vivo. Hum. Gene Ther. 4, 713-723.
36. SCHULTZ, E., ALBRIGHT, D.J., JARYSZAK, D.L., and DAVID, T.L. (1988). Survival of satellite cells in whole muscle transplants. Anat. Rec. 222, 12-17.
37. SHIRAFUJI, N., ASANO, S., MATSUDA, S., WATARI, K., TAKAKU, F., and NAGATA, S. (1989). A new bioassay for human granulocyte colony-stimulating factor (hG-CSF) using murine myeloblastic NSF-60 cells as targets and estimation of its levels in sera from normal healthy persons and patients with infectious and hematological disorders. Exp. Hematol. 17, 116-119.
38. SOUZA, L.M., BOONE, T.C., GABRILOVE, J., LAI, P.H., ZSEBO, K.M., MURDOCK, D.C., CHAZIN, V.R., BRUSZEWSKI, J., LU, H., CHEN, K.K., BARENDT, J., PLATZER, E., MOORE, M.A.S., MERTELSMANN, R., and WELTE, K. (1986). Recombinant human granulocyte colony-stimulating factor: Effects on normal and leukemic myeloid cells. Science 232, 61-65.
39. TANI, K., OZAWA, K., TAKAHASHI, T., OKANO, A., WATARI, K., MATSUDAIRA, T., TAJIKA, K., KARASUYAMA, H., NAGATA, S., ASANO, S., and TAKAKU, F. (1989). Implantation of fibroblasts transfected with human granulocyte colony-stimulating factor cDNA into mice as a model of cytokine-supplement gene therapy. Blood 74, 1274-1280.
40. THOMPSON, A.R. (1991). Status of gene transfer for hemophilia A and B. Thrombosis and Haemostasis 66, 119-122.
41. VITADELLO, M. (1994). Gene transfer in regenerating muscle. Hum. Gene Ther. 5, 11-18.
42. WELTE, K., ZEIDLER, C., REITER, A., MULLER, W., ODENWALD, E., SOUZA, L., and RIEHM, H. (1990). Differential effects of granulocyte-macrophage colony-stimulating factor and granulocyte colony-stimulating factor in children with severe congenital neutropenia. Blood 75, 1056-1063.
43. WOLFF, J.A., MALONE, R.W., WILLIAMS, P., CHONG, W., ACSADI, G., JANI, A., and FELGNER, P.L. (1990). Direct gene transfer into mouse muscle in vivo. Science 247, 1465-1468.
44. WRIGHT, W.E., BINDER, M., and FUNK, W. (1991). Cyclic amplification and selection of targets (CASTing) for the myogenin consensus binding site. Mol. Cell. Biol. 11, 4104-4110.
45. YABLONKA-REUVENI, Z., QUINN, L.S., and NAMEROFF, M. (1987). Isolation and clonal analysis of satellite cells from chicken pectoralis muscle. Dev. Biol. 119, 252-259.
46. YAO, S-N., and KURACHI, K. (1992). Expression of human factor IX in mice after injection of geneticallay modified myoblasts. Proc. Natl. Acad. Sci. USA 89, 3357-3361.
47. YAO, S-N., SMITH, K.J., and KURACHI, K. (1994). Primary myoblast-mediated gene transfer: persistent expression of human factor IX in mice. Gene Ther. 1, 99-107.